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# Krystal Biotech BLA for Topical Gene Therapy
- URL: https://www.fdaweb.com/krystal-biotech-bla-for-topical-gene-therapy/
- Published: 2022-06-22T12:00:00.000Z
- Updated: 2026-09-14T17:49:29.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5152121

Krystal Biotech has submitted a BLA for B-Vec (beremagene geperpavec) and its use in treating patients with dystrophic epidermolysis bullosa (DEB). The topical gene therapy is designed to treat DEB at the molecular level by providing the patient’s skin cells with two copies of the *COL7A1* gene to make functional COL7 protein, which addresses the underlying disease-causing mechanism, according to the company.

The submission is supported by data from two placebo controlled clinical trials — the GEM-3 trial ([NCT04491604](https://www.globenewswire.com/Tracker?data=NuwCVb8J-QlcGefxJyKKP0XyHpIJXYf3bCOVI%5Fkx3sjYbD8KEX3B0nnYof1LSSEW1xNkIPdrIW5dULNAN8eGHpFCoHPavoW79-fpdzbCWNx4BqjFkfkCGbkVVJbEmiqVcNKaiBtwpVib2Ye-U-e2yQ==&ref=fdaweb.com)) and the GEM-1/2 trial ([NCT03536143](https://www.globenewswire.com/Tracker?data=QvYwskSLwb4-yxHLXFhGrVoUu5GqBgeRyVHb8wF%5Fc-hY1NKYD7LwLh9V05g0pgPg4U6h2ukRECvnJeW6N5ovqx6uI8T4dMU4CSzPQGyy1u6DKDi4lA10ss%5FgOT7IuMlcAtfJFZY4d2MEfAHaILY4%5FQ==&ref=fdaweb.com)). The company says that in the GEM-3 trial, the primary endpoint of complete wound healing at six months was met and so was the secondary endpoint of complete wound healing at three-months. In GEM-1/2 trial, both mechanistic and clinical endpoints were met.